[Federal Register Volume 78, Number 127 (Tuesday, July 2, 2013)]
[Notices]
[Pages 39736-39737]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-15797]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2013-D-0576]
Draft Guidance for Industry: Considerations for the Design of
Early-Phase Clinical Trials of Cellular and Gene Therapy Products;
Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
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SUMMARY: The Food and Drug Administration (FDA) is announcing the
availability of a draft guidance document entitled ``Guidance for
Industry: Considerations for the Design of Early-Phase Clinical Trials
of Cellular and Gene Therapy Products'' dated July 2013. The draft
guidance document provides sponsors of Investigational New Drug
Applications (INDs) for cellular therapy (CT) and gene therapy (GT)
products (referred to collectively as CGT products) with
recommendations to assist in designing early-phase clinical trials of
CGT products.
DATES: Although you can comment on any guidance at any time (see 21 CFR
10.115(g)(5)), to ensure that the Agency considers your comment on this
draft guidance before it begins work on the final version of the
guidance, submit either electronic or written comments on the draft
guidance by November 22, 2013.
ADDRESSES: Submit written requests for single copies of the draft
guidance to the Office of Communication, Outreach and Development (HFM-
40), Center for Biologics Evaluation and Research (CBER), Food and Drug
Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 20852-
1448. Send one self-addressed adhesive label to assist the office in
processing your requests. The draft guidance may also be obtained by
mail by calling CBER at 1-800-835-4709 or 301-827-1800. See the
SUPPLEMENTARY INFORMATION section for electronic access to the draft
guidance document.
Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane,
rm. 1061, Rockville, MD 20852.
FOR FURTHER INFORMATION CONTACT: Melissa Reisman, Center for Biologics
Evaluation and Research (HFM-17), Food and Drug Administration, 1401
Rockville Pike, suite 200N, Rockville, MD 20852-1448, 301-827-6210.
[[Page 39737]]
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance document
entitled ``Guidance for Industry: Considerations for the Design of
Early-Phase Clinical Trials of Cellular and Gene Therapy Products,''
dated July 2013. The draft guidance document provides sponsors of INDs
for CGT products with recommendations to assist in designing early-
phase clinical trials of CGT products. The scope of this guidance is
limited to products for which the Office of Cellular, Tissue and Gene
Therapies/FDA has regulatory authority. CGT products within the scope
of this guidance meet the definition of ``biological product'' in
section 351(i) of the Public Health Service (PHS) Act (42 U.S.C.
262(i)). The guidance does not apply to those human cells, tissues, and
cellular-and tissue-based products (HCT/Ps) regulated solely under
section 361 of the PHS Act (42 U.S.C. 264), to products regulated as
medical devices under the Federal Food, Drug, and Cosmetic Act (the
FD&C Act), or to the therapeutic biological products for which the
Center for Drug Evaluation and Research (CDER) has regulatory
responsibility.
The design of early-phase clinical trials of CGT products often
differs from the design of clinical trials for other types of
pharmaceutical products. Differences in trial design are necessitated
by the distinctive features of these products, and also may reflect
previous clinical experience. The draft guidance document describes
features of CGT products that influence clinical trial design,
including product characteristics, manufacturing considerations and
preclinical considerations, and suggests other documents for additional
information. Consequently, the draft guidance document provides
recommendations with respect to these products as to clinical trial
design, including early-phase trial objectives, choosing a study
population, using a control group and blinding, dose selection,
treatment plans, monitoring and follow-up. Finally, the draft guidance
encourages prospective sponsors to meet with FDA review staff regarding
their IND submission and offers references for additional guidance on
submitting an IND.
The draft guidance is being issued consistent with FDA's good
guidance practices regulation (21 CFR 10.115). The draft guidance, when
finalized, will represent FDA's current thinking on this topic. It does
not create or confer any rights for or on any person and does not
operate to bind FDA or the public. An alternative approach may be used
if such approach satisfies the requirement of the applicable statutes
and regulations.
II. Paperwork Reduction Act of 1995
This draft guidance refers to previously approved collections of
information found in FDA regulations. These collections of information
are subject to review by the Office of Management and Budget (OMB)
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The
collections of information in 21 CFR part 312 have been approved under
OMB control number 0910-0014.
III. Comments
The draft guidance is being distributed for comment purposes only
and is not intended for implementation at this time. Interested persons
may submit either electronic comments regarding this document to http://www.regulations.gov or written comments to the Division of Dockets
Management (see ADDRESSES). It is only necessary to send one set of
comments. Identify comments with the docket number found in brackets in
the heading of this document. Received comments may be seen in the
Division of Dockets Management between 9 a.m. and 4 p.m., Monday
through Friday, and will be posted to the docket at http://www.regulations.gov.
IV. Electronic Access
Persons with access to the Internet may obtain the draft guidance
at either http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.
Dated: June 25, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-15797 Filed 7-1-13; 8:45 am]
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